EUCTR2018-001145-14-IT进行中（未招募）1 期
A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects inferior or equal to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) - ALD-104
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 35
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Informed consent is obtained from a competent custodial parent or guardian
- •with legal capacity to execute a local IRB/IEC approved consent. Informed
- •assent will be sought from capable subjects, in accordance with the directive
- •of the IRB/IEC and with local requirements.
- •2. Males aged 17 years and younger, at the time of parental/guardian consent
- •and, where appropriate, subject assent.
- •3. Active cerebral ALD as defined by:
- •a. Elevated VLCFA values, and
- •b. Active central nervous system (CNS) disease established by central
- •radiographic review of brain MRI demonstrating
- •i. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
- •ii. Gadolinium enhancement on MRI of demyelinating lesions.
- •4. Neurologic Function Score (NFS) = 1
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •2. Use of statins, Lorenzo’s Oil, or dietary regimens used to lower VLCFA levels.
- •Note: subjects must discontinue use of these medications at time of consent.
- •3. Receipt of an investigational study drug or procedure within 3 months before
- •Screening that might confound study outcomes. Use of investigational study drugs
- •is prohibited throughout the course of the study.
- •4. Any conditions that make it impossible to perform MRI studies (including allergies
- •to anesthetics or contrast agents).
- •5. Hematological compromise as evidenced by:
- •a. Peripheral blood ANC count < 1500 cells/mm3, and either
- •b. Platelet count < 100,000 cells/mm3 or
- •c. Hemoglobin < 10 g/dL.
- •6. Hepatic compromise as evidenced by:
- •a. Aspartate transaminase (AST) value >2.5 × ULN
- •b. Alanine transaminase (ALT) value >2.5 × ULN
- •c. Total bilirubin value >3.0 mg/dL, except if there is a diagnosis of
- •Gilbert’s Syndrome and the subject is otherwise stable
- •7. Baseline estimated glomerular filtration rate <70 mL/min/1.73 m2, as determined
- •using the Chronic Kidney Disease Epidemiology Collaboration creatinine equation
- •(see https://www.kidney.org/professionals/KDOQI/gfr_calculatorPed)
- •8. Cardiac compromise as evidenced by left ventricular ejection fraction <40%
- •9. Immediate family member with a known or suspected Familial Cancer Syndrome
- •(including but not limited to hereditary breast and ovarian cancer syndrome,
- •hereditary non-polyposis colorectal cancer syndrome, and familial adenomatous
- •polyposis).
- •10. Clinically significant uncontrolled, active bacterial, viral, fungal, parasitic, or
- •prionassociated
- •11. Positive for human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2); hepatitis B
- •virus (HBV); hepatitis C virus (HCV); human T lymphotrophic virus 1 (HTLV-1).
- •(Note that subjects who have been vaccinated against HBV [positive for HBV
- •surface antibodies] who are negative for other markers of prior HBV infection
- •[e.g., negative for HBV core Ab] are eligible. Subjects with past exposure to
- •HBV [HBcAb positive and/or HBeAb positive] are also eligible for the study
- •provided they have a negative test for HBV DNA. Also note that subjects who are
- •positive for anti-hepatitis C Ab are eligible as long as they have a negative hepatitis
- •C viral load).
- •12. Any clinically significant cardiovascular, hematological, or pulmonary
- •disease, or other disease or condition that would be contraindicated for any of the other study procedures..
- •13. Absence of adequate contraception for fertile subjects. Male subjects and their
- •female partners are required to use two different effective methods of contraception
- •from Screening through at least 6 months after Lenti-D Drug Product infusion. If
- •subjects are truly sexually abstinent (where true sexual abstinence is defined as
- •being in line with the preferred and usual lifestyle of the subject), no second method
- •is required.
- •14. Any contraindications to the use of G-CSF or plerixafor during the mobilization of
- •hematopoietic stem cells, and any contraindications to the use of busulfan or
- •fludarabine, including known hypersensitivity to the active substances or to any of the
- •excipients in their formulations.
- •15. Known hypersensitivity to protamine sulfate.
研究者
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